Abstract

Introduction

Regenerative medicine and particular adult stem cells represent an alternative option
with several fruitful therapeutic applications in patients suffering from chronic
lung diseases including idiopathic pulmonary fibrosis (IPF). Nevertheless, lack of
knowledge regarding the origin and the potential of mesenchymal stem cells (MSCs)
to differentiate into fibroblasts has limited their use for the treatment of this
dismal disease.

Patients and methods

To this end, we conducted a phase Ib, non-randomized, clinical trial to study the
safety of three endobronchial infusions of autologous adipose derived stromal cells
(ADSCs)-stromal vascular fraction (SVF) (0.5 million cells per kgr of body weight
per infusion) in patients with IPF (n=14) of mild to moderate disease severity (forced
vital capacity –FVC>50% predicted value and diffusion lung capacity for carbon monoxide-DLCO>35% of predicted value). Our primary end-point was incidence of treatment emergent
adverse events within 12 months. Alterations of functional, exercise capacity and
quality of life parameters at serial time points (baseline, 6 and 12 months after
first infusion) were exploratory secondary end-points.

Results

No cases of serious or clinically meaningful adverse events including short-term infusional
toxicities as well as long-term ectopic tissue formation were recorded in all patients.
Detailed safety monitoring through several time-points indicated that cell-treated
patients did not deteriorate in both functional parameters and indicators of quality
of life.